Apr 29 2014
Tekmira Pharmaceuticals Corporation, a leading developer of RNA interference (RNAi) therapeutics, today reported that Alnylam Pharmaceuticals, Inc. presented new clinical data for patisiran (ALN-TTR02), an RNAi therapeutic targeting transthyretin (TTR) in development for the treatment of TTR-mediated amyloidosis (ATTR), which is enabled by Tekmira's lipid nanoparticle (LNP) technology. The program represents the most clinically advanced application of Tekmira's proprietary LNP delivery technology.
"We are pleased with the progress reported today in Alnylam's patisiran development program, demonstrating sustained knockdown of serum TTR and tolerability with extended dosing, enabled by Tekmira's LNP technology. Our LNP delivery technology continues to drive the most significant RNAi advances in the field and enables multiple RNAi products in clinical development in a variety of therapeutic areas, including our own robust product pipeline. Today's announcement further demonstrates the promise of RNAi and validates our LNP technology," said Dr. Mark J. Murray, Tekmira's President and CEO.
Alnylam presented data at the International Symposium on Amyloidosis (ISA) held April 27 - May 1, 2014 in Indianapolis, Indiana. First, the company presented updated Phase 2 results in patients with Familial Amyloidotic Polyneuropathy (FAP) confirming robust TTR knockdown of up to 96% with a mean TTR knockdown of approximately 80%. It also presented preliminary results from the open-label extension (OLE) study with patisiran in patients that were enrolled in the Phase 2 study. Preliminary results from the OLE study showed that multiple doses of patisiran achieved sustained knockdown of serum TTR protein levels at the 80% target level through 168 days. Moreover, the OLE study results showed a favorable tolerability profile with up to eight doses administered. Finally, Alnylam presented results of a natural history, cross-sectional analysis study of 283 FAP patients aimed at measuring the rate of neuropathy progression and its correlation with disease severity. These results provide support for Alnylam's Phase 3 APOLLO trial where patisiran is being evaluated for its potential efficacy and safety in the FAP indication.
For more detailed information about the newly presented Phase II data for patisiran (ALN-TTR02), please refer to the Alnylam news release dated April 28, 2014 and the presentation of these data, which can be found on Alnylam's website at www.alnylam.com.
About RNAi and Tekmira's LNP
RNAi therapeutics have the potential to treat a broad number of human diseases by "silencing" disease causing genes. The discoverers of RNAi, a gene silencing mechanism used by all cells, were awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi therapeutics, such as "siRNAs," require delivery technology to be effective systemically. Tekmira believes its LNP technology represents the most widely adopted delivery technology for the systemic delivery of RNAi therapeutics. Tekmira's LNP platform is being utilized in multiple clinical trials by both Tekmira and its partners. Tekmira's LNP technology (formerly referred to as stable nucleic acid-lipid particles or SNALP) encapsulates siRNAs with high efficiency in uniform lipid nanoparticles that are effective in delivering RNAi therapeutics to disease sites in numerous preclinical models. Tekmira's LNP formulations are manufactured by a proprietary method which is robust, scalable and highly reproducible, and LNP-based products have been reviewed by multiple FDA divisions for use in clinical trials. LNP formulations comprise several lipid components that can be adjusted to suit the specific application.
About Tekmira
Tekmira Pharmaceuticals Corporation is a biopharmaceutical company focused on advancing novel RNAi therapeutics and providing its leading lipid nanoparticle delivery technology to pharmaceutical partners. Tekmira has been working in the field of nucleic acid delivery for over a decade and has broad intellectual property covering LNPs. Further information about Tekmira can be found at www.tekmira.com. Tekmira is based in Vancouver, B.C.
Source: http://www.tekmira.com